Introduction The medical treatment options for patients with Crohn’s disease (CD) are limited and patients resistant to those therapies are left requiring surgical operations that usually only achieve some symptomatic relief. Mesenchymal stem cells (MSC) have been shown to be effective for the treatment of CD, and we have demonstrated in animal experiments that human amnion-derived MSCs (AMSC) are a potential new therapeutic strategy. Therefore, we designed this study to investigate the safety and efficacy of AMSCs in patients with treatment-resistant CD.
Methods and analysis This is the protocol for an ongoing phase I/II, dual-centre, open-label, uncontrolled, dose–response study. The estimated enrolment is 6–12 patients with treatment-resistant, moderate CD. A dose of 1.0×106 cells/kg will be administered intravenously in the low-dose group at days 0 and 7. After confirming the safety of low-dose administration, a dose of 4.0×106 cells/kg will be administered intravenously in the high-dose group on days 0 and 7. The primary endpoint will measure the occurrence of adverse events related to acute infusion toxicity, and secondary endpoints will include long-term adverse events and efficacy of AMSC administration.
Ethics and dissemination The Institutional Review Board of Hokkaido University Hospital approved this study protocol (approval number H29-6). A report releasing study results will be submitted to an appropriate journal.
Discussion This study is the first to investigate the safety and efficacy of AMSC use for CD treatment. Our results will advance studies on more efficient and convenient methods to overcome the limits of available CD treatments.
Trial registration number UMIN000029841.
- Crohn’s disease
- stem cells
- bacterial interactions
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Contributors SOt contributed by writing the manuscript and in carrying out the trial. SOh is the principal investigator, responsible for conception, design, and processing of the trial. AM, HH, IK, and TI reviewed all protocol versions and contributed to the start-up of the trial. YMI contributed to the design of the trial and the statistical analysis plan. TK and SN contributed by reviewing all protocol versions and carrying out the trial. KY reviewed all protocol versions and was involved in manufacturing and quality control of AM01. NSat and NSak supervised and edited the protocol.
Funding This work was supported by the Research Project for Applications of Regenerative Medicine from the Japan Agency of Medical Research and Development (AMED, 17bk0104058h0002).
Competing interests None declared.
Patient consent Not required.
Ethics approval Institutional Review Board of Hokkaido University Hospital (approval number H29-6).
Provenance and peer review Not commissioned; externally peer reviewed.
Data sharing statement We will share the data after the trial is finished. Additional details of the study protocol can be requested from the corresponding author.